FDA Approves First Biologic to Treat Rare Clotting Disorder

The U.S. Food and Drug Administration (FDA) today licensed Ceprotin, the first biologic treatment for patients with a rare genetic defect that can cause a potentially life-threatening clotting disorder.

Ceprotin is made from the plasma of healthy human blood donors. It is a concentrated form of Protein C, a substance normally manufactured in the liver that circulates in the plasma in very small amounts. Protein C plays an important role in controlling blood coagulation by preventing the formation and growth of blood clots.

Severe congenital Protein C deficiency is a rare genetic defect found in one to two newborns for every million births. Patients with insufficient levels of Protein C experience abnormally high numbers of blood clots. Complete absence of the protein is fatal. Symptoms typically appear soon after birth. Clotting may occur in the blood vessels of the skin, eyes, brain, kidneys and throughout the body.

“This product offers much-needed treatment for the small number of patients with severe inherited Protein C deficiency,” said Jesse Goodman, M.D., M.P.H., director of FDA’s Center for Biologics Evaluation and Research. “If left untreated, clotting may result in blindness, severe brain damage, multi-organ failure and death for these patients.”

Patients with severe inherited Protein C deficiency must take oral or injected anticoagulant drugs on a regular basis to avoid blood clots. Ceprotin is intended to treat these patients when they are faced with a life-threatening situation from blood clots in the veins, or a severe skin and systemic blood clotting disorder known as Purpura fulminans.

The company enrolled all available patients for the pivotal trial. In 94 percent of the patients studied for Purpura fulminans, Ceprotin was found “effective.” In another 6 percent of patients, the treatment was found “effective with complications” because they required a dosage adjustment. Eighty percent of the treatments for blood clots in the veins were determined as “excellent” while the other 20 percent were determined as “good.”

The seven patients who took Ceprotin as a preventive measure before surgery or anticoagulation therapy experienced no blood clotting complications. Eight patients who were given Ceprotin as a long term preventive measure did not experience the severe skin and blood clotting events associated with Purpura fulminans.

The most common adverse reactions were rash, itching and lightheadedness.

FDA granted Ceprotin orphan drug status, which provides the manufacturer with financial incentives to develop a drug or biologic to treat a rare disease (affecting fewer than 200,000 people in the United States). Since 1983, more than 200 drugs and biological products have been brought to market in this way.

FDA reviewed the drug’s Biologics License Application under a priority review schedule.

Ceprotin is manufactured by Baxter Healthcare Corp. of Deerfield, Ill.